Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome

NCT02629991 | Completed Phase 2 Results DMC

• 1 other identifier: 14-10-427-01
• Study Type: interventional
• Target: 23
• Locations: 1 country
• Sites: 1

Brief Summary

The investigators propose a randomized double-blind 8 week treatment trial of intranasal oxytocin (IN-OXT) vs. placebo in 24 subjects aged 5 to 18 years with PWS in order to assess IN-OXT's affect on (1) Eating behaviors (2) Repetitive and disruptive behaviors and (3) Salivary OXT levels.

Conditions

Prader-Willi Syndrome; Hyperphagia

Keywords

Prader-Willi Syndrome (PWS); Hyperphagia

Outcome Measures

Primary Outcomes (4)

• Hyperphagia Questionnaire (HQ)- Total Score

  The Hyperphagia Questionnaire (HQ)- Total Score. The HQ is a 11-item clinician-rated instrument that was designed to measure food related behaviors in PWS. Items map onto 3 subscales; Hyperphagic Behavior, Hyperphagic Drive, and Hyperphagia Severity. The subscales are summed together to compute the Total Score. The Total Score ranges from 11-55, with higher scores indicating more hyperphagia and a worse outcome.

  Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.

• Hyperphagia Questionnaire (HQ)- Behavior Factor Score

  The Hyperphagia Questionnaire (HQ)- Behavior Factor Score assesses food seeking behaviors (e.g., stealing food). The Behavior Factor Score ranges from 5-25, with higher scores indicating more hyperphagic behaviors and a worse outcome.

  Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.

• Hyperphagia Questionnaire (HQ)- Drive Factor Score

  The Hyperphagia Questionnaire (HQ)- Drive Factor Score assesses the persistence in asking for food. The Drive Factor Score ranges from 4-20, with higher scores indicating higher hyperphagic drive and a worse outcome

  Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.

• Hyperphagia Questionnaire (HQ) -Severity Factor Score

  The Hyperphagia Questionnaire (HQ)- Severity Factor Score assesses the severity of hyperphagia. The Severity Factor Score ranges from 2-10, with higher scores indicating higher severity.

  Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.

Secondary Outcomes (6)

• Repetitive Behavior Scale-Revised (RBS-R) Ritualistic Behavior Factor Score

  Trends over multiple time points (Weeks 0, 4, and 8).

• Repetitive Behavior Scale-Revised (RBS-R) Sameness Factor Score

  Trends over multiple time points (Weeks 0, 4, and 8).

• Repetitive Behavior Scale-Revised (RBS-R) Stereotyped Behavior Factor Score

  Trends over multiple time points (Weeks 0, 4, and 8).

• Repetitive Behavior Scale-Revised (RBS-R) Compulsive Behavior Factor Score

  Trends over multiple time points (Weeks 0, 4, and 8).

• Repetitive Behavior Scale-Revised (RBS-R) Self-Injurious Factor Score

  Trends over multiple time points (Weeks 0, 4, and 8).

Study Arms (2)

Intranasal Oxytocin

EXPERIMENTAL

Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).

Drug: Intranasal Oxytocin (IN-OXT)

Matched Placebo

PLACEBO COMPARATOR

Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).

Other: Matched Placebo

Interventions

Intranasal Oxytocin (IN-OXT) DRUG

Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).

Also known as: Syntocinon

Intranasal Oxytocin

Matched Placebo OTHER

Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).

Matched Placebo

Eligibility Criteria

• Age: 5 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Male or Female child outpatients aged 5 to 18 years
• Diagnosis of PWS confirmed by genetic testing and patient medical records and history
• Stable pharmacologic, educational, behavioral and/or dietary interventions for 4 weeks prior to the study start, and for the duration of the study.
• Have a physical exam and laboratory results that are within the norms for PWS
• Have a parent/caregiver/guardian that is able to consent for their participation and complete assessments regarding the child's development and behavior improvement throughout the study.

You may not qualify if:

• Exposure to any investigational agent in the 30 days prior to randomization
• Prior chronic treatment with oxytocin.
• A primary psychiatric diagnosis other than ASD, including bipolar disorder, psychosis, schizophrenia, PTSD or major depressive disorder. These patients will be excluded due to potential confounding results.
• Pregnant or lactating patients. IN-OXT has not been studied in pregnant or lactating women.
• A medical condition that might interfere with the conduct of the study, confound interpretation of study results or endanger their own well-being.
• Plan to initiate or change nonpharmacologic or pharmacologic interventions during the course of the study.
• Females using an estrogen-based contraceptive. As an alternative to an estrogen based contraceptive, subjects will be counseled to use progesterone-based contraceptives; cervical caps; cervical sponges; or spermicidal foam in combination with a condom. Subjects will need to use a double barrier method to be in the study.

Sponsors & Collaborators

• Montefiore Medical Center: lead
• Foundation for Prader-Willi Research: collaborator

Study Sites (1)

Montefiore Medical Center, Albert Einstein College of Medicine

The Bronx, New York, 10467, United States

Location

Related Links

• Foundation for Prader Willi Research

MeSH Terms

Conditions

Prader-Willi Syndrome; Hyperphagia

Interventions

Oxytocin

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases; Signs and Symptoms, Digestive; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Posterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Bonnie Taylor
• Organization: Albert Einstein College of Medicine

botaylor@montefiore.org

7188397530

Study Officials

• Eric Hollander, MD

  Montefiore Medical Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: October 18, 2015
• First Posted: December 15, 2015
• Study Start: October 1, 2015
• Primary Completion: December 1, 2016
• Study Completion: May 1, 2018
• Last Updated: October 7, 2021
• Results First Posted: October 7, 2021

Record last verified: 2021-09

Locations

US (1)